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Investigation of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in Male Fragile X Syndrome Patients Aged 13-40 (IMPACT-FXS)

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ClinicalTrials.gov Identifier: NCT04823052
Recruitment Status : Recruiting
First Posted : March 30, 2021
Last Update Posted : July 18, 2022
Information provided by (Responsible Party):
Healx Limited

Brief Summary:
This study is to investigate the safety, tolerability and efficacy of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in males with Fragile X Syndrome (FXS) with confirmed full FMR1 mutation treated over a 10 week period in an outpatient setting.

Condition or disease Intervention/treatment Phase
Fragile X Syndrome Drug: Sulindac (HLX-0201), dose strength 1 Drug: Sulindac (HLX-0201), dose strength 2 Drug: Placebo Drug: Gaboxadol (HLX-0206) Phase 2

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Study Type : Interventional
Estimated Enrollment : 60 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Subjects will be randomly assigned to 1 of 4 treatment arms. All treatment arms will be conducted in parallel. Fifteen subjects will be included in each arm. The study treatment will be blinded to patients, carers and physicians.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2, Placebo-controlled, Adaptive Design Study to Explore the Safety and Efficacy of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) and Possible Other Treatments in Adolescent and Adult Males With Fragile X Syndrome (FXS)
Actual Study Start Date : May 25, 2022
Estimated Primary Completion Date : December 2022
Estimated Study Completion Date : December 2022

Resource links provided by the National Library of Medicine

Drug Information available for: Sulindac

Arm Intervention/treatment
Placebo Comparator: Placebo
One capsule, twice a day
Drug: Placebo
Placebo Capsule

Active Comparator: Sulindac (HLX-0201), dose strength 1
One capsule, twice a day
Drug: Sulindac (HLX-0201), dose strength 1
Sulindac (HLX-0201) Capsule

Active Comparator: Sulindac (HLX-0201), dose strength 2
One capsule, twice a day
Drug: Sulindac (HLX-0201), dose strength 2
Sulindac (HLX-0201) Capsule

Active Comparator: Gaboxadol (HLX-0206)
One capsule, twice a day
Drug: Gaboxadol (HLX-0206)
Gaboxadol (HLX-0206) Capsule

Primary Outcome Measures :
  1. NIH Cognitive Toolbox [ Time Frame: Day 70 ]
  2. Clinical Global Impression - I [ Time Frame: Day 70 ]
  3. Aberrant Behavior Checklist [ Time Frame: Day 70 ]
  4. Anxiety, Depression, and Mood Scale [ Time Frame: Day 70 ]
  5. FXS Domain Specific Concerns [ Time Frame: Day 70 ]
    The Clinician/Caregiver FXS Domain Specific Concerns allows for the subject specific symptoms of concern to be assessed on an ongoing basis throughout the study. The specific concerns that correlate to the 6 domains (Clinician) or 3 Domains (Caregiver) will be assessed using a 7-point Likert scale.

Secondary Outcome Measures :
  1. To assess the safety and tolerability of each dose [ Time Frame: Day 70 ]
    Incidence of adverse events (AEs) and serious adverse events (SAEs) reported during the study. Change from baseline to Day 70 in physical examinations and assessment of vital signs.

  2. Kiddie Test of Attentional Performance (KiTAP) [ Time Frame: Day 70 ]
  3. Emotional Faces Tobii Eye Tracking [ Time Frame: Day 70 ]
  4. EEG [ Time Frame: Day 70 ]
    Resting state whole brain EEG spectra activity in 3 EEG assessments (EEG spectra, auditory evoked response potential (ERP) and Chirp modulated sweep.

  5. CGI-S [ Time Frame: Day 70 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   13 Years to 40 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Subject consents to participate, or if the subject are not the subjects own legal guardian, offers assent supported by legally authorized representative consent. Caregiver also commits to the study requirements prior to any study-related procedures
  • Willing and able to comply with the study procedures as specified in the protocol and to comply with the study drug administration
  • Subject and caregiver are both able to understand the spoken national language clearly and caregiver can read and write to complete study assessments
  • Males aged 13 to 40 years (inclusive)
  • Has FXS with molecular genetic confirmation of the full FMR1 mutation (>200 CGG repeats). May have been confirmed historically or at Screening
  • Weight ≥45 kg
  • CGI-S score ≥4
  • Is in general good health as deemed by the Investigator, determined by physical examination, medical history and laboratory tests
  • If receiving sertraline, is on a stable, well-tolerated dose for the previous 3 months with no further changes anticipated
  • Agrees not to discuss treatment outcomes on social media until subject has completed their End of Therapy visit

Exclusion Criteria:

  • Active or history of peptic or gastric ulcer or hemorrhage
  • Any chronic major medical comorbid condition deemed by the Investigator as presenting added risk to the subject, including but not limited to refractory hypertension, kidney disease, or liver disease
  • Diagnosed with diabetes (Type 1 or II) or receiving any anti-diabetic medication
  • Unstable seizure disorder defined by any seizure within 6 months prior to baseline visit and/or a change in any anti-convulsant drug dosing in the 60 days prior to study entry
  • Patients with cardiovascular disease risk factors: Uncontrolled high blood pressure (systolic blood pressure >150 mmHg), Unstable angina, History of documented myocardial infarction or cerebrovascular accident, NYHA Class III and IV heart failure, Known uncontrolled hyperlipidemia as LDL-C ≥190 mg/dL or triglycerides ≥ 500 mg/dL
  • Chronic use of NSAIDs or other anti-inflammatory agents
  • Currently taking or have taken any cannabidiol (CBD) preparation within 30 days prior to screening
  • Currently taking or have taken sulindac or gaboxadol within 30 days prior to screening
  • Currently taking GABAergic agents (i.e., acamprosate, baclofen, vigabatrin, tiagabine, riluzole, benzodiazepines, and gabapentin)
  • Changes in psychotropic or anti-convulsant (where taken for reasons other than seizure control) drug treatment within 30 days prior to Screening
  • Significant changes in any educational, behavioral and/or dietary interventions the month prior to Screening
  • Planned initiation of new, or modification of ongoing, interventions during the study
  • History of adverse effects of sulindac or other NSAIDs that would prevent safe study completion
  • Unable or unwilling to take oral medication (whole capsule) or history of dysphagia or malabsorption
  • Has abnormal baseline laboratory assessments including, but not limited to, ALT or AST or total bilirubin >1.5 × ULN, or other clinically relevant laboratory abnormality
  • Has a clinically significant abnormal ECG, heart rate or BP at Screening as judged by the Investigator
  • Has received an investigational drug (either approved or not approved) in any prior clinical study within 30 days or 5 half-lives (whichever is longer) prior to Screening

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04823052

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United States, Colorado
Children's Hospital Colorado Not yet recruiting
Aurora, Colorado, United States, 80045
Contact: Hannah Fleming    720-777-2137    Hannah.Fleming@childrenscolorado.org   
Principal Investigator: Nicole Tartaglia         
United States, Florida
University of Miami Not yet recruiting
Miami, Florida, United States, 33136
Contact: Yaneysy Penate    646-527-1042    yxp303@med.miami.edu   
Principal Investigator: Deborah Barbouth         
United States, Georgia
Emory University School of Medicine Not yet recruiting
Atlanta, Georgia, United States, 30322
Contact: Jean Luan McColl    404-778-8570    jean.luan@emory.edu   
Principal Investigator: Amy Talboy         
United States, Illinois
Rush University Medical Center & Children's Hospital Recruiting
Chicago, Illinois, United States, 60612
Contact: Loren Escot    312-942-4036    Loren_Escot@rush.edu   
Principal Investigator: Elizabeth Berry-Kravis         
United States, Maryland
Kennedy Krieger Insitute Not yet recruiting
Baltimore, Maryland, United States, 21205
Contact: Nadia Moore    443-923-7619    MooreNa@kennedykrieger.org   
Principal Investigator: Dejan Budimirovic         
United States, Massachusetts
University of Massachusetts Chan Medical School Recruiting
Worcester, Massachusetts, United States, 10655
Contact: Taylor Merk    774-455-4100    Childresearch@umassmed.edu   
Principal Investigator: Jean Frazier         
United States, New York
Icahn School of Medicine (Mount Sinai) Not yet recruiting
New York, New York, United States, 10029
Contact: Karli Hedstrom    212-659-1450    karli.hedstrom@mssm.edu   
Principal Investigator: Reymundo Lozano         
Australia, Queensland
Children's Health Queensland Hospital and Health Service Not yet recruiting
Brisbane, Queensland, Australia, 4101
Contact: Michael Duhig    07 3068 2593    Michael.Duhig@health.qld.gov.au   
Principal Investigator: Honey Heussler         
Australia, Victoria
Fragile X Alliance Clinic Not yet recruiting
Caulfield, Victoria, Australia, 3161
Contact: Jonathan Cohen       jcohen@travelclinic.com.au   
Principal Investigator: Jonathan Cohen         
Murdoch Children's Research Institute Not yet recruiting
Melbourne, Victoria, Australia, 3052
Contact: Kylie Crompton       kylie.crompton@mcri.edu.au   
Principal Investigator: David Amor         
Sponsors and Collaborators
Healx Limited
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Principal Investigator: Elizabeth Berry-Kravis Rush University Medical Center & Children's Hospital
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Responsible Party: Healx Limited
ClinicalTrials.gov Identifier: NCT04823052    
Other Study ID Numbers: HLX002-0201
First Posted: March 30, 2021    Key Record Dates
Last Update Posted: July 18, 2022
Last Verified: July 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Healx Limited:
Fragile X Syndrome
Additional relevant MeSH terms:
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Fragile X Syndrome
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Sex Chromosome Disorders
Chromosome Disorders
Congenital Abnormalities
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System
Anti-Inflammatory Agents, Non-Steroidal
Analgesics, Non-Narcotic
Sensory System Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Inflammatory Agents
Antirheumatic Agents
Antineoplastic Agents
Cyclooxygenase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action